BARRY J. BYRNE, M.D., Ph.D.

Dr. Byrne is an professor of Pediatrics. As a member of the Graduate Faculty and Department of Molecular Genetics and Microbiology and Gene Therapy Center, he is actively involved in laboratory research.

The laboratory is involved in developing new genetic therapies for cardiovascular disease. In the area of cardiomyopathy, we are studying gene replacement in an autosomal recessive form of fatal cardiomyopathy in children. The disease is the prototype of lysosomal storage disorders leading to skeletal and cardiac muscle weakness.

We have used AAV vectors to achieve sustained correction of the gene deficiency and correction of the phenotype in natural and transgenic mouse models of the disease. The current therapy is being propsed for human clinical trials. Similar therapies are being used to combat cardiac transplantation rejection.

Secondly, we are investigating the ability of mesenchymal stem cells to undergo myocardial specification of the purpose of tissue repair in the heart. Finally, several projects are focused on the use of AAV vectors injected into striated muscle to achieve sustained release of therapeutic proteins, including thrombolytic factors and coagulation factors.

These projects are currently funded by the the NIH, AHA and foundation grants.